Lundbeck's amlenetug receives orphan drug status in Japan for MSA

Lundbeck, a global pharmaceutical company, has successfully obtained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare for its human monoclonal antibody, amlenetug, intended for the treatment of multiple system atrophy (MSA). This recognition is an addition to the previous accolades the company has received, which include the SAKIGAKE designation in Japan and ODDs from both the US Food and Drug Administration and the European Medicines Agency.

Amlenetug is designed to target and bind with all major forms of extracellular α-synuclein, a protein linked to MSA. The drug aims to inhibit the uptake and prevent the aggregation and seeding of this protein. The antibody's active Fc region could also boost the immune system's ability to clear α-synuclein/mAb complexes through microglial uptake. This antibody was developed through a collaborative research and licensing agreement between Lundbeck and the Danish biotechnology company Genmab.

Lundbeck has initiated a randomized, parallel-group Phase III clinical trial named MASCOT, to evaluate the safety and efficacy of amlenetug in the treatment of MSA. This significant trial will take place across multiple continents including Asia, Australia, Europe, and North America. The study is structured in two segments. During the double-blind phase, participants will receive either high or low doses of amlenetug or a placebo over a period of 72 weeks. This will be followed by an open-label phase, where all participants will have access to the antibody treatment.

The primary focus of the MASCOT trial is to thoroughly assess the safety, efficacy, and tolerability of the antibody. The administration of the drug will occur via intravenous infusion every four weeks. Johan Luthman, Lundbeck's head of research and development and executive vice-president, expressed optimism about the ODD recognition in Japan, emphasizing the potential of amlenetug to serve as a viable treatment for individuals suffering from MSA. Luthman also noted the promise of amlenetug in potentially slowing the clinical progression of this debilitating disease and showed eagerness to advance its development through the MASCOT trial.

In addition to these developments, Lundbeck has been actively working to bolster its position in the neuro-rare disease market. In December 2024, the company completed the acquisition of all outstanding shares of Longboard Pharmaceuticals. This strategic move underscores Lundbeck's commitment to expanding its expertise and resources in addressing neuro-rare diseases, thereby reinforcing its status as a leader in this specialized field.

Through these efforts, Lundbeck continues to demonstrate its dedication to advancing scientific research and providing innovative treatments for neuro-rare diseases like MSA. The ongoing developments in the MASCOT trial, combined with the company's strategic acquisitions, highlight Lundbeck's commitment to improving the lives of patients affected by these challenging conditions. As the global medical community awaits the results from the MASCOT trial, there is a sense of hope that amlenetug could offer a meaningful therapeutic option for MSA patients, potentially altering the course of this devastating disease.